A Phase I Feasibility And Safety Study of Fluorescein-Specific (FITC-E2) CAR T Cells In Combination With Parenterally Administered Folate-Fluorescein (UB-TT170) For Osteogenic Sarcoma

NCT05312411 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: ENLIGHTen-01
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to see if a new treatment could help patients who have osteosarcoma that does not go away with treatment (is refractory) or comes back after treatment (is recurrent).This study is testing a combination of study therapies, UB-TT170 and genetically modified chimeric antigen receptor T lymphocyte (CAR T) cells, which work together in a way that is different from chemotherapy. In this study, researchers will take some of your blood and remove the T cells in a process called "apheresis". Then the T cells are taken to a lab and changed to CAR T cells that recognize the flags from UB-TT170. Once researchers think they have grown enough CAR T cells, called antiFL(FITC-E2) CAR T cells, to fight your cancer, you may get some chemotherapy to make room in your body for the new cells and then have those cells put back in your body. A few days after the you get your CAR T cell infusion you will start to get infusions of UB-TT170, with the dose slowly increasing for the first few infusions until you have reached a maximum dose that you will get on a regular schedule. The UB-TT170 will attach to your tumor cells and flag them so that they attract the CAR T cells. When the CAR T cells see the labeled tumor cells they can kill the tumor cells. The active part of the study lasts about 8 months, and if you get the CAR T cell infusion you will be in long-term follow-up for 15 years.

Conditions

Osteosarcoma

Keywords

osteosarcoma; bone cancer; pediatric sarcoma; young adult sarcoma

Outcome Measures

Primary Outcomes (1)

• Adverse events associated with ex-vivo expanded autologous T cells genetically modified to express an antiFL(FITC-E2) CAR administered with UB-TT170 will be assessed

  The type, frequency, severity, and duration of adverse events will be summarized

  30 days

Secondary Outcomes (2)

• Ability to manufacture antiFL(FITC-E2) CAR cells

  28 Days

• Evaluate the pharmacokinetics of UB-TT170 in combination with the anti-FL(FITC-E2) CAR T cells

  25 days

Study Arms (1)

UB-TT170 following SCRI-E2CAR_EGFrtv1

EXPERIMENTAL

Following CAR T cell administration, subjects will receive a first Course of 3 escalating doses of UB-TT170 over 2 weeks followed by fixed weekly dosing for 2 weeks. If eligible, subjects may proceed to Courses 2 - 4 consisting of 7 weekly doses of UB-TT170.

Biological: SCRI-E2CAR_EGFRtv1; Drug: UB_TT170

Interventions

SCRI-E2CAR_EGFRtv1 BIOLOGICAL

Autologous CD4+ and CD8+ T cells that have been genetically modified to express antiFL(FITC-E2)

UB-TT170 following SCRI-E2CAR_EGFrtv1

UB_TT170 DRUG

Bispecific small molecule adapter formulated with phosphate buffered saline

UB-TT170 following SCRI-E2CAR_EGFrtv1

Eligibility Criteria

• Age: 15 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Refractory or recurrent/progressive osteosarcoma that has failed first line therapy for Osteosarcoma per NCCN or upfront Children's Oncology Group clinical trial and is not amenable to surgical resection (must meet one of the following):
• New site of measurable disease by radiographic imaging or histologic confirmation
• New site of evaluable disease by radiographic imaging (including FDG-PET) or histologic confirmation
• Greater than 20% increase in at least one tumor dimension documented by CT/MRI, AND a maximum absolute increase of 5 mm in longest dimension of existing lesion(s) (previously irradiated lesions may be included)
• Persistent measurable disease or FDG-PET avid bone metastasis that has failed to achieve complete remission to upfront conventional therapy (surgery, radiotherapy and/or chemotherapy)
• Able to tolerate apheresis, including placement of temporary apheresis catheter, if necessary, or already has an apheresis product available for use in manufacturing
• Life expectancy ≥ 8 weeks
• Lansky or Karnofsky score ≥ 50
• Anti-cancer agents, radiotherapy, cytoxic chemotherapy, biologic therapy, anti-tumor antibody therapy, genetically modified cell therapy, and, if no apheresis product available, corticosteroid therapy (excluding physiologic replacement), discontinued within protocol specified wash-out period
• Adequate hematologic, renal, hepatic, cardiac, and respiratory function.
• Negative HIV, hepatitis B and C test within 3 months
• If of child-bearing or fathering potential, willing to use highly effective contraception through 12 months following final stud drug infusion

You may not qualify if:

• Active malignancy other than primary malignant solid tumor diagnosis (CNS intracranial metastases are allowed)
• Ongoing, symptomatic CNS pathology requiring medical intervention
• Receiving external beam radiotherapy
• Presence of active, severe infection
• Primary immunodeficiency syndrome
• Pregnant or breast feeding
• Unwilling to provide consent/assent for study participation, including 15 year follow up
• Presence of any condition that, in the opinion of the investigator, would prohibit the subject from undergoing treatment under this protocol.

Sponsors & Collaborators

• Seattle Children's Hospital: lead
• Umoja Biopharma: collaborator

Study Sites (1)

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

MeSH Terms

Conditions

Osteosarcoma; Bone Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Sarcoma; Neoplasms by Site; Bone Diseases; Musculoskeletal Diseases

Study Officials

• Catherine Albert, MD

  Seattle Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical Director, Seattle Children's Therapeutics

Study Record Dates

• First Submitted: March 20, 2019
• First Posted: April 5, 2022
• Study Start: May 20, 2022
• Primary Completion: March 12, 2025
• Study Completion (Estimated): May 1, 2040
• Last Updated: November 20, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)